Development and validation of a patient-reported outcome in systemic sclerosis: the Hand scleroDerma lived Experience (HAnDE) scale.
Journal
The British journal of dermatology
ISSN: 1365-2133
Titre abrégé: Br J Dermatol
Pays: England
ID NLM: 0004041
Informations de publication
Date de publication:
01 2022
01 2022
Historique:
accepted:
05
08
2021
pubmed:
7
8
2021
medline:
4
3
2022
entrez:
6
8
2021
Statut:
ppublish
Résumé
Hand involvement in systemic sclerosis (SSc) is at the core of the disease, with a substantial impact on both functional aspects and quality of life. There is no patient-reported outcome (PRO) scale globally assessing hand involvement in SSc. To develop and validate a PRO scale, the Hand scleroDerma lived Experience (HAnDE) scale, to assess the lived experience of hand involvement in patients with SSc. This was an exploratory sequential mixed-methods study with two phases: (i) PRO development through an inductive process to analyse the structure of lived experience, involving 21 patients with SSc; and (ii) PRO validation by assessing the psychometric properties of the scale among 105 patients with SSc. Phase 1 enabled us to generate the 18-item provisional scale. From Phase 2, the mean (SD) total score of the scale was 29·16 (16·15). The item reduction process retained 16 items with five levels of answers (range 0-64). Internal consistency of the 16-item version was excellent (Cronbach's alpha = 0·946). Construct validity was very good, principal component analysis pointing towards a unidimensional instrument, with one factor explaining 56% of the variance, and concurrent validity being confirmed: Cochin Hand Function Scale r = 0·66; Health Assessment Questionnaire - Disability index r = 0·58; Hospital Anxiety and Depression Scale, anxiety r = 0·51, depression r = 0·4; Mouth Handicap in Systemic Sclerosis scale r = 0·61; 36-Item Short Form Health Survey, physical component r = -0·48, mental component r = -0·46; and Kapandji score r = -0·46. The correlations were statistically significant (P < 0·05). We propose, for future trials and clinical practice in SSc, a new PRO, the HAnDE scale, that assesses all the dimensions - functional, aesthetic, relational, existential and emotional - of the lived experience of hand involvement in SSc.
Sections du résumé
BACKGROUND
Hand involvement in systemic sclerosis (SSc) is at the core of the disease, with a substantial impact on both functional aspects and quality of life. There is no patient-reported outcome (PRO) scale globally assessing hand involvement in SSc.
OBJECTIVES
To develop and validate a PRO scale, the Hand scleroDerma lived Experience (HAnDE) scale, to assess the lived experience of hand involvement in patients with SSc.
METHODS
This was an exploratory sequential mixed-methods study with two phases: (i) PRO development through an inductive process to analyse the structure of lived experience, involving 21 patients with SSc; and (ii) PRO validation by assessing the psychometric properties of the scale among 105 patients with SSc.
RESULTS
Phase 1 enabled us to generate the 18-item provisional scale. From Phase 2, the mean (SD) total score of the scale was 29·16 (16·15). The item reduction process retained 16 items with five levels of answers (range 0-64). Internal consistency of the 16-item version was excellent (Cronbach's alpha = 0·946). Construct validity was very good, principal component analysis pointing towards a unidimensional instrument, with one factor explaining 56% of the variance, and concurrent validity being confirmed: Cochin Hand Function Scale r = 0·66; Health Assessment Questionnaire - Disability index r = 0·58; Hospital Anxiety and Depression Scale, anxiety r = 0·51, depression r = 0·4; Mouth Handicap in Systemic Sclerosis scale r = 0·61; 36-Item Short Form Health Survey, physical component r = -0·48, mental component r = -0·46; and Kapandji score r = -0·46. The correlations were statistically significant (P < 0·05).
CONCLUSIONS
We propose, for future trials and clinical practice in SSc, a new PRO, the HAnDE scale, that assesses all the dimensions - functional, aesthetic, relational, existential and emotional - of the lived experience of hand involvement in SSc.
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
96-105Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2021 British Association of Dermatologists.
Références
Denton CP, Khanna D. Systemic sclerosis. Lancet 2017; 390:1685-99.
Erol K, Gok K, Cengiz G, Ozgocmen S. Hand functions in systemic sclerosis and rheumatoid arthritis and influence on clinical variables. Int J Rheum Dis 2018; 21:249-52.
Jewett LR, Hudson M, Malcarne V et al. Sociodemographic and disease correlates of body image distress among patients with systemic sclerosis. PLoS ONE 2012; 7:e33281.
Bretterklieber A, Painsi C, Avian A et al. Impaired quality of life in patients with systemic sclerosis compared to the general population and chronic dermatoses. BMC Res Notes 2014; 7:594.
Hudson M, Thombs BD, Steele R et al. Quality of life in patients with systemic sclerosis compared to the general population and patients with other chronic conditions. J Rheumatol 2009; 36:768-72.
Almeida C, Almeida I, Vasconcelos C. Quality of life in systemic sclerosis. Autoimmun Rev 2015; 14:1087-96.
Jewett LR, Razykov I, Hudson M et al. Prevalence of current, 12-month and lifetime major depressive disorder among patients with systemic sclerosis. Rheumatology (Oxford) 2013; 52:669-75.
Kallen MA, Mayes MD, Kriseman YL et al. The symptom burden index: development and initial findings from use with patients with systemic sclerosis. J Rheumatol 2010; 37:1692-8.
Deshpande PR, Rajan S, Sudeepthi BL, Abdul Nazir CP. Patient-reported outcomes: a new era in clinical research. Perspect Clin Res 2011; 2:137-44.
Barsotti S, Orlandi M, Codullo V et al. One year in review 2019: systemic sclerosis. Clin Exp Rheumatol 2019; 37 (Suppl. 119):S3-14.
Ingegnoli F, Carmona L, Castrejon I. Systematic review of systemic sclerosis-specific instruments for the EULAR Outcome Measures Library: an evolutional database model of validated patient-reported outcomes. Semin Arthritis Rheum 2017; 46:609-14.
Pauling JD, Frech TM, Domsic RT, Hudson M. Patient participation in patient-reported outcome instrument development in systemic sclerosis. Clin Exp Rheumatol 2017; 35 (Suppl. 106):184-92.
FDA. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. December 2009. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-support-labeling-claims (last accessed 5 September 2021).
Creswell JW, Klassen AC, Plano Clark VL, Smith KC, for the Office of Behavioral and Social Sciences Research. Practices for Mixed Methods Research in the Health Sciences. Bethesda, MD: National Institutes of Health, 2013; 541-5.
van den Hoogen F, Khanna D, Fransen J et al. 2013 classification criteria for systemic sclerosis: an American College of Rheumatology/European League Against Rheumatism collaborative initiative. Arthritis Rheum 2013; 65:2737-47.
Sibeoni J, Verneuil L, Manolios E, Révah-Levy A. A specific method for qualitative medical research: the IPSE (Inductive Process to analyze the Structure of lived Experience) approach. BMC Med Res Methodol 2020; 20:216.
Rannou F, Poiraudeau S, Berezné A et al. Assessing disability and quality of life in systemic sclerosis: construct validities of the Cochin Hand Function Scale, Health Assessment Questionnaire (HAQ), systemic sclerosis HAQ, and Medical Outcomes Study 36-item Short Form Health Survey. Arthritis Rheum 2007; 57:94-102.
Mouthon L, Rannou F, Bérezné A et al. Development and validation of a scale for mouth handicap in systemic sclerosis: the Mouth Handicap in Systemic Sclerosis scale. Ann Rheum Dis 2007; 66:1651-5.
Poole JL, Steen VD. The use of the Health Assessment Questionnaire (HAQ) to determine physical disability in systemic sclerosis. Arthritis Care Res 1991; 4:27-31.
Cossutta R, Zeni S, Soldi A et al. [Evaluation of quality of life in patients with systemic sclerosis by the SF-36 questionnaire]. Reumatismo 2002; 54:122-7 (in Italian).
Zigmond AS, Snaith RP. The Hospital Anxiety and Depression Scale. Acta Psychiatr Scand 1983; 67:361-70.
Keitel W, Hoffman H, Weber G, Krieger U. [Development of a arm functional test for rheumatologic diseases]. Dtsch Gesundheitsw 1971; 26:1901-3 (in German).
R Core Team. R: A Language and Environment for Statistical Computing. Vienna, Austria: R Foundation for Statistical Computing. Available at: www.r-project.org/ (last accessed 10 September 2021).
Nunnally JC, Bernstein IH. Psychometric Testing, 3rd edn. New York: McGraw-Hill, 1994.
Cohen J. Statistical Power Analysis for the Behavioral Sciences, 2nd edn. Hillsdale, NJ: Lawrence Erlbaum Associates, Inc., 1988; 75-107.
Pauling JD, Domsic RT, Saketkoo LA et al. Multinational qualitative research study exploring the patient experience of Raynaud’s phenomenon in systemic sclerosis. Arthritis Care Res (Hoboken) 2018; 70:1373-84.
Hughes M, Allanore Y, Chung L et al. Raynaud phenomenon and digital ulcers in systemic sclerosis. Nat Rev Rheumatol 2020; 16:208-21.
Hughes M, Pauling JD, Jones J et al. Multicenter qualitative study exploring the patient experience of digital ulcers in systemic sclerosis. Arthritis Care Res (Hoboken) 2020; 72:723-33.
Sumpton D, Thakkar V, O’Neill S et al. “It’s not me, it’s not really me”. Insights from patients on living with systemic sclerosis: an interview study. Arthritis Care Res (Hoboken) 2017; 69:1733-42.
Stamm TA, Mattsson M, Mihai C et al. Concepts of functioning and health important to people with systemic sclerosis: a qualitative study in four European countries. Ann Rheum Dis 2011; 70:1074-9.
Poole JL, Gallegos M, O’Linc S. Reliability and validity of the Arthritis Hand Function Test in adults with systemic sclerosis (scleroderma). Arthritis Care Res 2000; 13:69-73.
Steen VD, Medsger TA Jr. The value of the Health Assessment Questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis patients over time. Arthritis Rheum 1997; 40:1984-91.
Gholizadeh S, Meier A, Malcarne VL. Measuring and managing appearance anxiety in patients with systemic sclerosis. Expert Rev Clin Immunol 2019; 15:341-6.
Gholizadeh S, Fox RS, Mills SD, Jewett LR, Thombs BD, Malcarne VL. Coping with the disfigurement of scleroderma: facial, skin, and hand changes. In: Scleroderma: From Pathogenesis to Comprehensive Management (Varga J, Denton C, Wigley FM, Allanore Y, Kuwana M, eds), 2nd edn. New York: Springer International Publishing, 2017; 713-21.
Maddali-Bongi S, Del Rosso A, Mikhaylova S et al. Impact of hand and face disabilities on global disability and quality of life in systemic sclerosis patients. Clin Exp Rheumatol 2014; 32 (Suppl. 86):S-15-20.
Mills SD, Kwakkenbos L, Carrier M-E et al. Validation of the Social Appearance Anxiety Scale in patients with systemic sclerosis: a scleroderma patient-centered intervention network cohort study. Arthritis Care Res (Hoboken) 2018; 70:1557-62.
Sołowiej-Chmiel J, Sierakowska M. Functioning in the emotional sphere and ways of coping with chronic connective tissue disease. Reumatologia 2018; 56:321-7.
Bérezné A, Seror R, Morell-Dubois S et al. Impact of systemic sclerosis on occupational and professional activity with attention to patients with digital ulcers. Arthritis Care Res (Hoboken) 2011; 63:277-85.
Milette K, Thombs BD, Dewez S et al. Scleroderma patient perspectives on social support from close social relationships. Disabil Rehabil 2020; 42:1588-98.
Bruni C, Raja J, Denton CP, Matucci-Cerinic M. The clinical relevance of sexual dysfunction in systemic sclerosis. Autoimmun Rev 2015; 14:1111-5.
Rosato E, Rossi C, Molinaro I et al. Sexual distress, sexual dysfunction and relationship quality in women with systemic sclerosis: correlation with clinical variables. Int J Immunopathol Pharmacol 2014; 27:279-85.
Bragazzi NL, Watad A, Gizunterman A et al. The burden of depression in systemic sclerosis patients: a nationwide population-based study. J Affect Disord 2019; 243:427-31.
Faezi ST, Paragomi P, Shahali A et al. Prevalence and severity of depression and anxiety in patients with systemic sclerosis: an epidemiologic survey and investigation of clinical correlates. J Clin Rheumatol 2017; 23:80-6.
Newton EG, Thombs BD, Groleau D. The experience of emotional distress among women with scleroderma. Qual Health Res 2012; 22:1195-206.
Young A, Namas R, Dodge C, Khanna D. Hand impairment in systemic sclerosis: various manifestations and currently available treatment. Curr Treatm Opt Rheumatol 2016; 2:252-69.
Hughes M, Pauling JD. Exploring the patient experience of digital ulcers in systemic sclerosis. Semin Arthritis Rheum 2019; 48:888-94.
Lasch KE, Marquis P, Vigneux M et al. PRO development: rigorous qualitative research as the crucial foundation. Qual Life Res 2010; 19:1087-96.
Mouthon L, Poiraudeau S, Vernon M et al. Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument. Arthritis Res Ther 2020; 22:3.
Foocharoen C, Tyndall A, Hachulla E et al. Erectile dysfunction is frequent in systemic sclerosis and associated with severe disease: a study of the EULAR Scleroderma Trial and Research group. Arthritis Res Ther 2012; 14:R37.
Schouffoer A, van der Marel J, Ter Kuile MM et al. Impaired sexual function in women with systemic sclerosis: a cross-sectional study. Arthritis Rheum 2009; 61:1601-8.
Mouthon L, Alami S, Boisard AS et al. Patients’ views and needs about systemic sclerosis and its management: a qualitative interview study. BMC Musculoskelet Disord 2017; 18:230.
Sacristán JA, Dilla T, Dıáz-Cerezo S et al. Patient-physician discrepancy in the perception of immune-mediated inflammatory diseases: rheumatoid arthritis, psoriatic arthritis and psoriasis. A qualitative systematic review of the literature. PLoS ONE 2020; 15:e0234705.
Tong A, Sainsbury P, Craig J. Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. Int J Qual Health Care 2007; 19:349-57.